公开(公告)号：US12298313B1

公开(公告)日：2025-05-13

申请号：US19013863

申请日：2025-01-08

Applicant: Genzyme Corporation

Inventor： Xiaoying Jin ,  Catherine O'Riordan ,  Lin Liu ,  Kate Zhang

IPC: G01N33/68 ,  C12N15/86 ,  G01N30/72 ,  G01N30/02

Abstract: Provided herein are methods for determining the serotype of a virus particle and/or or determining the heterogeneity of a virus particle (e.g., an AAV particle). In other embodiments, the invention provides methods to determine the heterogeneity of AAV particles. In some aspects, the invention provides viral particles (e.g., rAAV particles) with improved stability and/or improved transduction efficiency by increasing the acetylation and/or deamidation of capsid proteins.

公开(公告)号：US10760079B2

公开(公告)日：2020-09-01

申请号：US16566565

申请日：2019-09-10

Applicant: Genzyme Corporation

Inventor： Lisa M. Stanek ,  Adam Palermo ,  Brenda Richards ,  Sergio Pablo Sardi ,  Catherine O'Riordan ,  Antonius Song

IPC: C07H21/04 ,  C12N15/113

Abstract: Provided herein are RNAi molecules including a first strand containing a guide sequence and a second strand comprising a non-guide sequence where the non-guide sequence contains a bulge opposite the seed region of the guide sequences; e.g., opposite the cleavage sequence. In some aspects, the invention provides RNAi for treating Huntington's disease. Further provided herein are expression cassettes, vectors (e.g., rAAV, recombinant adenoviral, recombinant lentiviral, and recombinant HSV vectors), cells, viral particles, and pharmaceutical compositions containing the RNAi. Yet further provided herein are methods and kits related to the use of the RNAi, for example, to treat Huntington's disease.

公开(公告)号：US10450563B2

公开(公告)日：2019-10-22

申请号：US15549895

申请日：2016-02-09

Applicant: Genzyme Corporation

Inventor： Lisa M. Stanek ,  Adam Palermo ,  Brenda Richards ,  Sergio Pablo Sardi ,  Catherine O'Riordan ,  Antonius Song

IPC: C07H21/04 ,  C12N15/113

Abstract: Provided herein are RNAi molecules including a first strand containing a guide sequence and a second strand comprising a non-guide sequence where the non-guide sequence contains a bulge opposite the seed region of the guide sequences; e.g., opposite the cleavage sequence. In some aspects, the invention provides RNAi for treating Huntington's disease. Further provided herein are expression cassettes, vectors (e.g., rAAV, recombinant adenoviral, recombinant lentiviral, and recombinant HSV vectors), cells, viral particles, and pharmaceutical compositions containing the RNAi. Yet further provided herein are methods and kits related to the use of the RNAi, for example, to treat Huntington's disease.

公开(公告)号：US12123880B2

公开(公告)日：2024-10-22

申请号：US18321542

申请日：2023-05-22

Applicant: Genzyme Corporation

Inventor： Xiaoying Jin ,  Catherine O'Riordan ,  Lin Liu ,  Kate Zhang

IPC: G01N33/68 ,  C12N15/86 ,  G01N30/72 ,  G01N30/02

CPC classification number: G01N33/6848 ,  C12N15/86 ,  G01N30/72 ,  C12N2750/14123 ,  C12N2750/14143 ,  G01N2030/027

Abstract: Provided herein are methods for determining the serotype of a virus particle and/or or determining the heterogeneity of a virus particle (e.g., an AAV particle). In other embodiments, the invention provides methods to determine the heterogeneity of AAV particles. In some aspects, the invention provides viral particles (e.g., rAAV particles) with improved stability and/or improved transduction efficiency by increasing the acetylation and/or deamidation of capsid proteins.

公开(公告)号：US20210261625A1

公开(公告)日：2021-08-26

申请号：US17162356

申请日：2021-01-29

Applicant: GENZYME CORPORATION

Inventor： Amy Frederick ,  Xiaoying Jin ,  Lin Liu ,  Catherine O'Riordan ,  Jennifer Sullivan

IPC: C07K14/005 ,  C12N15/86

Abstract: Provided herein are modified adeno-associated viral (AAV) capsid proteins, compositions (e.g., rAAV) comprising the capsid proteins, and nucleic acids encoding the capsid proteins. The AAV capsids provided herein confer retinal cell tropism and/or corneal cell tropism, and mediate improved transduction efficiency in clinically relevant ocular cell types such as photoreceptors and/or corneal endothelial cells. Also provided are nucleic acids encoding the capsid proteins, and AAV particles comprising the capsid proteins.

公开(公告)号：US10383953B2

公开(公告)日：2019-08-20

申请号：US15127757

申请日：2015-03-20

Applicant: Genzyme Corporation

Inventor： Catherine O'Riordan ,  Matthew Adamowicz

IPC: A61K48/00 ,  A61K31/7105 ,  C12N15/86 ,  A61K9/00 ,  C12N15/113 ,  A61K38/17

Abstract: Provided herein are methods for treating retinitis pigmentosa using an AAV particles encoding miR-708. In one aspect, viral particles are administered to the eye of a human subject; for example, by subretinal injection. Viral particles comprising AAV5 capsids or mutants thereof are contemplated.

公开(公告)号：US12201698B2

公开(公告)日：2025-01-21

申请号：US17383052

申请日：2021-07-22

Applicant: Genzyme Corporation

Inventor： Catherine O'Riordan ,  Matthew Adamowicz

IPC: A61K48/00 ,  A61K9/00 ,  A61K31/7105 ,  A61K38/17 ,  C12N15/113 ,  C12N15/86

Abstract: Provided herein are methods for treating retinitis pigmentosa using an AAV particles encoding miR-708. In one aspect, viral particles are administered to the eye of a human subject; for example, by subretinal injection. Viral particles comprising AAV5 capsids or mutants thereof are contemplated.

公开(公告)号：US20240384271A1

公开(公告)日：2024-11-21

申请号：US18626784

申请日：2024-04-04

Applicant: Genzyme Corporation

Inventor： Seng Cheng ,  Sarah Melissa Jacobo ,  Takako Moriguchi ,  Catherine O'Riordan ,  Guoxiang Ruan

IPC: C12N15/113 ,  A61K45/06 ,  A61P21/00 ,  C12N15/86

Abstract: Provided herein are RNAi molecules for treating myotonic dystrophy type 1 (DM1). Further provided herein are expression cassettes, vectors (e.g., rAAV), viral particles, and pharmaceutical compositions containing the RNAi. Yet further provided herein are methods and kits related to the use of the RNAi, for example, to treat DM1.

公开(公告)号：US20230365968A1

公开(公告)日：2023-11-16

申请号：US18296283

申请日：2023-04-05

Applicant: Genzyme Corporation

Inventor： Seng Cheng ,  Sarah Melissa Jacobo ,  Takako Moriguchi ,  Catherine O'Riordan ,  Guoxiang Ruan

IPC: C12N15/113 ,  C12N15/86 ,  A61P21/00

CPC classification number: C12N15/113 ,  C12N15/86 ,  A61P21/00 ,  C12N2750/14143

Abstract: Provided herein are RNAi molecules for treating myotonic dystrophy type 1 (DM1). Further provided herein are expression cassettes, vectors (e.g., rAAV), viral particles, and pharmaceutical compositions containing the RNAi. Yet further provided herein are methods and kits related to the use of the RNAi, for example, to treat DM1.

公开(公告)号：US20250027951A1

公开(公告)日：2025-01-23

申请号：US18801293

申请日：2024-08-12

Applicant: Genzyme Corporation

Inventor： Xiaoying JIN ,  Catherine O'Riordan ,  Lin Liu ,  Kate Zhang

IPC: G01N33/68 ,  C12N15/86 ,  G01N30/02 ,  G01N30/72

Abstract: Provided herein are methods for determining the serotype of a virus particle and/or or determining the heterogeneity of a virus particle (e.g., an AAV particle). In other embodiments, the invention provides methods to determine the heterogeneity of AAV particles. In some aspects, the invention provides viral particles (e.g., rAAV particles) with improved stability and/or improved transduction efficiency by increasing the acetylation and/or deamidation of capsid proteins.